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基因组编辑重新定义了心血管领域的精准医学。

Genome Editing Redefines Precision Medicine in the Cardiovascular Field.

作者信息

Dzilic Elda, Lahm Harald, Dreßen Martina, Deutsch Marcus-André, Lange Rüdiger, Wu Sean M, Krane Markus, Doppler Stefanie A

机构信息

Department of Cardiovascular Surgery, German Heart Center Munich, Technische Universität München, Lazarettstraße 36, 80636 Munich, Germany.

Insure (Institute for Translational Cardiac Surgery), Department of Cardiovascular Surgery, German Heart Center, Technische Universität München, Lothstraße 11, 80636 Munich, Germany.

出版信息

Stem Cells Int. 2018 Mar 14;2018:4136473. doi: 10.1155/2018/4136473. eCollection 2018.

Abstract

Genome editing is a powerful tool to study the function of specific genes and proteins important for development or disease. Recent technologies, especially CRISPR/Cas9 which is characterized by convenient handling and high precision, revolutionized the field of genome editing. Such tools have enormous potential for basic science as well as for regenerative medicine. Nevertheless, there are still several hurdles that have to be overcome, but patient-tailored therapies, termed precision medicine, seem to be within reach. In this review, we focus on the achievements and limitations of genome editing in the cardiovascular field. We explore different areas of cardiac research and highlight the most important developments: (1) the potential of genome editing in human pluripotent stem cells in basic research for disease modelling, drug screening, or reprogramming approaches and (2) the potential and remaining challenges of genome editing for regenerative therapies. Finally, we discuss social and ethical implications of these new technologies.

摘要

基因组编辑是一种强大的工具,可用于研究对发育或疾病至关重要的特定基因和蛋白质的功能。最近的技术,尤其是以操作简便和高精度为特点的CRISPR/Cas9,彻底改变了基因组编辑领域。此类工具在基础科学以及再生医学方面具有巨大潜力。然而,仍有几个障碍需要克服,但被称为精准医学的针对患者定制的疗法似乎已触手可及。在本综述中,我们重点关注基因组编辑在心血管领域的成就和局限性。我们探索心脏研究的不同领域,并突出最重要的进展:(1)基因组编辑在人类多能干细胞用于疾病建模、药物筛选或重编程方法的基础研究中的潜力,以及(2)基因组编辑用于再生疗法的潜力和尚存的挑战。最后,我们讨论这些新技术的社会和伦理影响。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b7c8/5872631/97e45d815aef/SCI2018-4136473.001.jpg

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